Genetic therapies have the potential to work for any person with CF, regardless of their CFTR mutations. This includes people who don't qualify for CFTR modulators or can't take them for any other reason.
Read our quick guide to learn how these potential treatments could work for any person with CF, including people with nonsense and other rare mutations.
Several types of genetic therapies are being developed for CF, including mRNA therapy, gene therapy, and gene editing, and they each come with different benefits and challenges. Keep reading for more in-depth information about our genetic therapy research.
mRNA therapy is one way to deliver the correct genetic instructions to cells, which would allow them to make functional CFTR protein regardless of an individual’s CF mutations.
Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF.
Gene editing is a way to make a permanent change to the DNA inside of a cell. That means gene editing therapies could provide lasting – potentially curative – benefits from just one or a few treatments.
For genetic therapies to work in cystic fibrosis, specifically engineered DNA or RNA molecules need to get inside the cells of the lung or other organs affected by CF. The process of getting these molecules into cells is referred to as delivery.
Stem cells are a special type of cell that can divide and produce many other cell types. Scientists are studying how adult stem cells could be used to treat cystic fibrosis.